Personalized Medicine Meets the Real World

THE ERA OF PERSONALIZED medicine was heralded long before its arrival. But not until Herceptin (trastizumab) came onto the market in 1998 to treat breast cancer in HER-2 positive patients did companies understand just how much genomics could alter the healthcare landscape.

Since then, companies have advanced the science, and have even brought a few tailor-made therpies to market. NitroMed, for example, figured out that BiDil (isosorbide/hydralazine) works best in African Americans. But while pharma has pushed this and other personalized medicines through clinical development, much of the data that proves these drugs' utility in real-world settings can't be obtained through the traditional route of experimental medicine. Instead, payers are demanding clinical and cost-effectiveness information for these very expensive medicines, as well as long-term safety and efficacy information. So great is the need for coverage of these pricey, but often life-saving, therapies, that it is changing the traditional research paradigm.

Biology of an informed Consent Form

Now, the industry is looking toward observational research to fill in the gaps. Observational research includes epidemiology, the scientific study of the distribution and determinants of disease, and outcomes research, which evaluates the status of participants after receiving care. Both of these disciplines look at patients and drugs outside of controlled lab settings. Instead of dictating practice, researchers observe, record, and build a body of evidence that shows how drugs are used, whether they work, and if so, in which patients. Pharma no longer controls all aspects of treatment, but instead relies on scientists who know how to design and analyze data collected in non-experimental settings like doctors' offices.

Certainly, there is an urgent need for this type of research. Pharma's pipelines are filled with personalized medicines, and private and public payers are hinging their coverage on the all-important value claims this research is uniquely positioned to address. However, most companies are still in the thick of clinical development, and aren't thinking about the observational research they will need to demonstrate value to buyers. This article helps executives get out in front, by outlining the challenges personalized medicine poses to the research framework and offers advice on how to design research programs to ensure maximum uptake of the product.

Intrinsic Challenges

The very nature of personalized medicine challenges the way scientists normally handle drug development. There are some unique issues that characterize research for tailor-made therapies.

The right patients are hard to find Even large disease categories look like a collection of rare diseases when patients are grouped based on common polymorphisms and biomarkers. For example, although lung cancer is the leading cancer killer of both US men and women, Iressa (gefitinib) and Tarceva (erlotinib) are most successful when given to a subset of just 10 percent of lung cancer patients with a particular set of mutations in their epidermal growth-factor receptors.

Although advances in technology for examining DNA, proteins, and other biomarkers allow companies to understand and describe these patient subgroups, genetic testing is not yet a widespread or standard part of most physicians' armatorium. As such, many patients who can be helped by personalized medicines are simply not recognized.

Informed consent is more challenging The Office of Human Research Protections (OHRP) and the Office of Civil Rights (OCR) offer confusing and, at times, conflicting information to guide research practices with respect to informed consent and privacy. However, researchers of genomic medicines have the added burden of requirements outlined by the Centers for Disease Control, which don't necessarily conform to those required by OHRP and OCR.